Anybody else noticed that not a peep out of parents of the boys in the Phase 3 trial? I don't think it's a bad thing, but I also don't think it's a coincidence. My hunch is that the company has asked them to remain silent while the trial is ongoing, in order to not seem as if they are manipulating the FDA. Could be wrong, but it makes sense, and it's a strategy I agree with.
Awww, too bad, Biomarin. Could it be that the EMA realizes that eteplirsen is better and wants to hedge their decision by waiting to see more data from etep?
No, they don't need him. It was probably agreed to in order to avoid a lawsuit. He's consulting and that's it. You can be sure he will have no part of day-to-day issues. And frankly, you'd better pray the company lets him nowhere near the FDA.
No. Two boys went non-ambulatory shortly after the trial started. Two boys began to noticeably decline at the last update. The issue is that eteplirsen can only do so much with muscles that are already damaged. So I won't be surprised if all of the boys eventually end up non-ambulatory. To date, however, eteplirsen seems to have done a remarkable job of keeping muscles with little to no deterioration healthy (i.e., upper body including heart).
Eteplirsen is not a cure; nor has it ever been one. But the earlier the boys begin receiving it, the better their chances for a long life.
It's also certainly different from what GSK said. So who are you going to believe? The company that dumped drisapersen because of, among other things, its toxicity? Or the company that has a financial incentive to blow off drisapersen's negative effects?
Use your head for once.
Check Adam Feuerstein's newly posted article. These dates suggest a back-to-back review for drisa and etep, which is what many of us thought, and what one in particular insisted wouldn't happen.
This, in my opinion, is a bigger deal than Marburg/Ebola speculation, and the stock's strength has nothing to do with either of those things. It's because the market is shifting its opinion on eteplirsen's chances for early approval. Anything else is just a side show at this point. Eyes on the prize. Loving the 180-degree about-face in this stock compared to last year!
Of course I know the difference. I presume that you are aware of these things:
1. An effective vaccine greatly reduces the urgency for finding a curative treatment because it will prevent widespread outbreaks.
2. Sarepta's Ebola treatment--despite the company making it widely known its current supply was available for testing--was never tested, and the government expressed no interest in doing so.
3. Sarepta's Ebola treatment is very expensive to make.
4. There are several other companies with treatments already being tested.
So knowing these things, a quick yes or no question for you or anyone insistent on believing SRPT will make money from Ebola:
Will Sarepta's Ebola treatment produce profit for the company?
Sarepta has incredible potential, but Ebola won't be part of its platform. Email management and ask them if you need verification.
This guy was bearish before as well. He's clearly a paid hack working for a fund. I highly doubt he's even a doctor. I mean, he's bullish on MNKD for goodness sake.
Definitely a possibility that there are 1-2 new non-ambulatory boys, but the spin on this being similar to natural history is just flat out false. Even if this is the case, they have done much better than the natural history of DMD suggests.
Generally agree with you, but...
1. There will not be any 24-week efficacy data because there is no way to prove efficacy at 24 weeks. That was true of the P2 as well, so I'm not sure why you think that P3 will somehow have efficacy data that early. 36 weeks is needed to show efficacy.
2. Unless the FDA accepts dystrophin as a surrogate marker--and that is very questionable--it would be a regular approval, meaning other exons would still have to go through the same rigorous trials. If dystrophin is accepted, then it could be an accelerated approval and open up the door to quickly approving eteplirsen for other exons.
Look brutha, here's the thing. You don't even need to be bullish on Sarepta to realize that this stock is absolutely ripe for a run-up, and it will happen. You really don't need to be a genius to figure that out. Sorry for calling you offensive names. I feel as if I should have offered a hug instead. You seem really upset with SRPT's gains.
I'm as bullish on Sarepta as I've ever been, but your suggestion that they don't need an Adcom is silly. Yes, it's pretty clear that the drug works, but the fact remains that the P2 trial did only have 12 boys. It would be irresponsible to approve any drug for a previously untreatable disease without having an expert panel review and vote on the drug. That's just absurd to suggest otherwise and doing so would open the door for all kinds of #$%$ drugs to try and do the same. The FDA has never done what you are suggesting, nor will they ever do what you are suggesting. They should have acted quicker with eteplirsen, but that doesn't mean they should give AA without a thorough review and Adcom.
Correct. Although interestingly enough, the FDA shortly thereafter removed the dates and changed them to "to be determined." I'm guessing they realized that they unintentionally tipped their hand regarding SRPT's NDA.
I think you are probably correct regarding press releases, and I'm sure this data will appear in publications as well. But we will see this information outside of publications and probably sooner than it will appear in those publications. The data you are referencing will be included in the briefing documents for the Adcom, and they will be made available to the public three business days before the Adcom convenes.